FDA

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FDAs Critical Path Initiative

• Kathryn M. Carbone, M.D.
• Assoc. Director for Research, CBER, FDA
• www.fda.gov/oc/initiatives/criticalpath.htm

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Tomorrows Medical Breakthroughs

• Many serious diseases afflict our population and
  are waiting for better treatments autism,
  addictive disorders, Alzheimer's disease, AIDS,
  bipolar disorders, cancer, cystic fibrosis, heart
  diseases, diabetes, morbid obesity, multiple
  sclerosis, muscular dsytrophy, rheumatoid
  arthritis, osteoarthritis, systemic lupus,
• schizophrenia, stroke, and many more

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Three Dimensions of the Critical Path

• Assessment of Safety how to predict if a
  potential product will be harmful?
• Preclinical assays
• Clinical trial design
• Proof of Efficacy -- how to determine if a
  potential product will have medical benefit?
• Biomarkers, e.g., Surrogate markers of efficacy
• Industrialization how to manufacture a product
  at commercial scale with consistently high
  quality?
• Predict scale up problems early
• Quality Standards Assays

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Proposal FDA Proactively Improving the Product
Development Process

• Apply new science to improve predictability in
  product development, not just scientific
  discovery
• Scientific knowledge and scientific tools to more
  efficiently assess candidate products

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Critical Path Research Outcome of Improving
Prediction

• Reduce cost by focusing resources on the best
  candidate products early in development
• More quantitative, less empiric, more predictable

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FDA The Critical Path Initiative

• Bringing attention and focus to the need for
  targeted scientific efforts to modernize the
  techniques and methods used to evaluate the
  safety, efficacy and quality of medical products
  as they move from product selection and design to
  mass manufacture.

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Why FDA? Unique Role of the Agency vis-a-vis the
Critical Path

• FDA scientists are involved in review during
  product development--they see the successes,
  failures, and missed opportunities
• FDA guidance documents are based on science and
  foster innovation and improve chances of success.
• Convening and coordinating role for new biomarker
  and clinical method development

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ROLE OF FDA IN THE PROCESS
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Examples of Opportunities with FDA Products

• Develop and make available well characterized
  cell banks (and related methods to assay for
  safety/adventitious agents) useful for vaccine
  and other biologics production
• Characterization of cell therapies links to
  standardized outcomes (e.g. HPSCs)
• Methods validation of pathogen inactivation for
  blood, plasma, tissues and other products
• Multipathogen and rapid detection methodologies
  for biologics including blood and tissue products
• Improving longevity/storage of blood, blood
  products and tissues
• Others as determined by working with stakeholders